基于证实残疾进展时间和多发性硬化症不规则观察的比较有效性的方法
Thomas Pa Debray1,2, Gabrielle Simoneau3, Massimiliano Copetti4
1Julius Centrum voor Gezondheidswetenschappen en Eerstelijns Geneeskunde, Utrecht, Netherlands.
Statistical methods in medical research
|June 12, 2023
概括
多层次的多重归算准确地分析了在不规则时间收集的真实世界数据. 这种方法改善了治疗效果的估计和对多发性硬化症疾病修饰疗法的置信区间覆盖率.
科学领域:
- 生物统计学 生物统计学
- 现实世界的数据分析分析.
- 纵向数据建模 纵向数据建模
背景情况:
- 现实世界数据 (RWD) 能够在临床实践中进行比较治疗有效性研究.
- RWD通常具有选择性结果记录和不规则的测量时间,使分析复杂化.
- 现有的归算方法可能无法捕捉纵向轨迹或充分处理缺失.
研究的目的:
- 建议扩展多层次的多重归算,用于分析具有不规则观察时间的RWD.
- 在多发性硬化症 (MS) 病例研究中评估这一新型归算方法的性能.
- 为了比较多层次的多次归算与标准的单次归算技术.
主要方法:
- 开发了一种针对不规则时间的纵向结果量身定制的多层次多重归算的扩展.
- 应用该方法来分析MS患者使用扩展残疾状况表 (EDSS) 数据确认残疾进展的时间.
- 进行了模拟研究,以评估与单次归算相比,偏差和置信区间覆盖率.
主要成果:
- 多层次的多重归算产生了较少偏差的治疗效果估计.
- 拟议的方法证明了信任区间覆盖率的改善.
- 即使没有随机 (MNAR) 结果,也观察到有效性.
结论:
- 多级多重归算是分析纵向RWD与不规则采样的一种可靠方法.
- 这种方法提高了在现实环境中对治疗比较的可靠性.
- 该方法为了解多发性硬化症等疾病的疾病轨迹和治疗效果提供了有价值的工具.
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